Therapeutic Outcomes and Electrophysiological Biomarkers in Anti-Myelin-Associated Glycoprotein Neuropathy: A Multicenter Cohort Study in South Korea

Min, Young Gi; Han, Hee-Jo; Shin, Ha Young; Baek, Jong-Gyu; Kim, Jun-Soon; Park, Kyung-Seok; Baek, Seol-Hee; Yoo, Ilhan; Hu, So-Young; Kwon, Young Nam; Choi, Seok-Jin; Kim, Sung-Min; Hong, Yoon-Ho; Sung, Jung-Joon

doi:10.3988/jcn.2023.0127

Cited by 3 publications

(1 citation statement)

References 54 publications

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“…86 Another BTK inhibitor, zanubrutinib, was recently reported as potentially effective in anti-MAG neuropathy. 87 A Phase II single-arm open-label trial with zanubrutinib in combination with rituximab is currently ongoing for anti-MAG related polyneuropathy (MAGNAZ) in the Netherlands (NCT05939037).…”

Section: Bruton Tyrosine Kinase Inhibitorsmentioning

confidence: 99%

Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Current Therapeutic Approaches and Future Outlooks

Rajabally

2024

ITT

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Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a treatable autoimmune disorder, for which different treatment options are available. Current first-line evidence-based therapies for CIDP include intravenous and subcutaneous immunoglobulins, corticosteroids and plasma exchanges. Despite lack of evidence, cyclophosphamide, rituximab and mycophenolate mofetil are commonly used in circumstances of refractoriness and, more debatably, of perceived overdependence on first-line therapies. Rituximab is currently the object of a randomized controlled trial for CIDP. Based on case series, and although rarely considered, haematopoietic autologous stem cell transplants may be effective in refractory disease, with low mortality and high remission rates. A new therapeutic option has appeared with efgartigimod, a neonatal Fc receptor blocker, recently shown to significantly lower relapse rate versus placebo, after withdrawal from previous immunotherapy. Other neonatal Fc receptor blockers, nipocalimab and batoclimab, are under study. The C1 complement-inhibitor SAR445088, acting in the proximal portion of the classical complement system, is currently the subject of a new study in treatment-responsive, refractory and treatment-naïve subjects. Finally, Bruton Tyrosine Kinase inhibitors, which exert anti-B cell effects, may represent another future research avenue. The widening of the therapeutic armamentarium enhances the need for improved evaluation of treatment effects and reliable biomarkers in CIDP.

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Section: Bruton Tyrosine Kinase Inhibitorsmentioning

confidence: 99%

Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Current Therapeutic Approaches and Future Outlooks

Rajabally

2024

ITT

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Neuropathy with anti-myelin-associated glycoprotein antibodies: update on diagnosis, pathophysiology and management

Min,

Visentin,

Briani

et al. 2024

J Neurol Neurosurg Psychiatry

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Antimyelin-associated glycoprotein (MAG) neuropathy is a rare autoimmune demyelinating peripheral neuropathy caused by IgM autoantibodies targeting MAG. The typical presentation is that of a slowly progressive, distal, length-dependent, predominantly sensory, sometimes ataxic neuropathy, frequently accompanied by upper limb tremor. Distal motor weakness may subsequently occur. The clinical presentation may vary and rarely be consistent with that of typical chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), as well as have an aggressive and rapidly disabling course. The diagnosis of anti-MAG neuropathy is based on the detection of anti-MAG antibodies through ELISA or western blot analysis, primarily in presence of an IgM monoclonal gammopathy. Anti-MAG neuropathy may occur without or with haematological malignancy. Electrophysiology is characteristic of a predominantly distal demyelinating neuropathy. Intravenous immunoglobulins and plasma exchange have unproven benefits, but may provide short-term effects. Cytotoxic therapies are commonly used, although without an evidence base. Rituximab, an anti-B-cell monoclonal antibody was studied in two randomised controlled trials, neither of which achieved their primary outcome. However, a meta-analysis of these two studies demonstrated improvement of disability at 8–12 months. A recent trial with lenalidomide was interrupted prematurely due to a high rate of venous thromboembolism. There are currently two ongoing trials with Bruton’s tyrosine kinase inhibitors. Symptom control is otherwise frequently needed. Outcome measures used for other inflammatory neuropathies present limitations in anti-MAG neuropathy. International registries such as the planned IMAGiNe study may, in future, provide answers to the many remaining questions.

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Favorable long-term outcomes of autoimmune nodopathy with mycophenolate mofetil

Min,

Ju,

Sung

2024

Front. Neurol.

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Autoimmune nodopathy (AN) is a rare immune-mediated neuropathy characterized by autoantibodies against nodal or paranodal proteins. Patients with AN generally respond poorly to immunoglobulin therapy, and as a newly defined condition, there are currently no established treatment guidelines. Although rituximab shows potential as a therapeutic option, its high cost, limited availability, and the need for infusion monitoring hinder its use as a first-line treatment in many countries. In this report, we identified AN antibodies in five of 106 serum samples (4.7%) prospectively collected from patients initially diagnosed with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP): anti-neurofascin 155 (NF155) in 2 patients, anti-contactin-1 (CNTN1) in 1, anti-contactin associated protein 1 (CASPR1), and anti-NF186/140 in 1. Notably, we observed favorable long-term outcomes in these patients following treatment with mycophenolate mofetil (MMF) and corticosteroids. Given that these patients had not responded to immunoglobulin therapy and/or experienced relapses with corticosteroid monotherapy in their prior episodes, we propose MMF as a cost-effective treatment strategy for AN.

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Therapeutic Outcomes and Electrophysiological Biomarkers in Anti-Myelin-Associated Glycoprotein Neuropathy: A Multicenter Cohort Study in South Korea

Cited by 3 publications

References 54 publications

Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Current Therapeutic Approaches and Future Outlooks

Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Current Therapeutic Approaches and Future Outlooks

Neuropathy with anti-myelin-associated glycoprotein antibodies: update on diagnosis, pathophysiology and management

Favorable long-term outcomes of autoimmune nodopathy with mycophenolate mofetil

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